Natural history

Clene Reports Significant Survival Benefit With CNM-Au8® Treatment in ALS EAP Compassionate Use Programs

Retrieved on: 
Thursday, February 22, 2024
ACT, Massachusetts General Hospital, Mass, Master of Science, NINDS, Bangladesh Technical Education Board, Neuromuscular disease, ALS, CNM, Doctor of Philosophy, NIH, FDA, Record, Hospital, Risk, Natural history, EAPS, Multiple sclerosis, Safety, Data, Patient, Survival, Disease, Harvard Medical School, Columbia University, EAP, Mortality, DSM-IV codes, Pharmaceutical industry

CNM-Au8 30 mg treatment was well-tolerated, without a single serious adverse event attributed to CNM-Au8, and no significant safety findings reported.

Key Points: 
  • CNM-Au8 30 mg treatment was well-tolerated, without a single serious adverse event attributed to CNM-Au8, and no significant safety findings reported.
  • A pooled survival analysis of EAP participants treated with CNM-Au8 30 mg was compared to two independent datasets derived from PRO-ACT and the ALS/MND Natural History Consortium.
  • The EAP dataset was comprised of 256 participants with ALS of which 220 EAP participants had all baseline values available for matching.
  • The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Clene supported the first EAP (EAP01) launched in 2019.

Thermo Fisher Scientific Launches CorEvitas Clinical Registry in Generalized Pustular Psoriasis

Retrieved on: 
Friday, March 8, 2024
Health, Health Technology, Other Science, General Health, Clinical Trials, Research, Science, Therapy, Incidence, Natural history, Woman, GPP, Registry, Quality of life, RWE, Thermo Fisher Scientific, Prevalence, PPD, Rash, Patient, Flare, Autoimmunity, Yale University, Disease

Thermo Fisher Scientific Inc., the world leader in serving science, today announced the launch of a new CorEvitas syndicated clinical registry in generalized pustular psoriasis (GPP).

Key Points: 
  • Thermo Fisher Scientific Inc., the world leader in serving science, today announced the launch of a new CorEvitas syndicated clinical registry in generalized pustular psoriasis (GPP).
  • This registry, which is open to enrollment, is CorEvitas’ 10th syndicated disease registry and addresses an unmet need for real-world evidence (RWE) related to the clinical and patient-reported outcomes of patients with GPP.
  • Thermo Fisher acquired CorEvitas, a leading provider of gold-standard real-world evidence solutions, in August 2023.
  • The registry was launched with clinical guidance from two leading GPP clinical experts who serve as scientific advisors: Dr. Mark Lebwohl, dean for clinical therapeutics, Icahn School of Medicine at Mount Sinai; and Dr. Bruce Strober, clinical professor, Department of Dermatology at Yale University.

Two Year Sustained Cognitive Benefits of Hydromethylthionine Mesylate (HMTM) Indicated by TauRx's LUCIDITY Trial

Retrieved on: 
Thursday, March 7, 2024
Mental Health, Neurology, Clinical Trials, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Blood, Natural history, University, Alzheimer's disease, Multimedia, Research, Pathology, News, ADAS, Conference, AFL, Patient, Disease, Psychiatry, ADNI, Dementia, MRI, Brain, Pharmaceutical industry

View the full release here: https://www.businesswire.com/news/home/20240307066794/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240307066794/en/
    New 24-month data show sustained benefits across the disease spectrum from early to moderate dementia.
  • Analyses comparing the study participants to closely matched real world data and meta-analytical controls, showed significantly reduced disease progression in participants in the LUCIDITY trial.
  • In the early disease subgroup there was a significant reduction in transition to the dementia stage of AD.
  • Its secondary pharmacological action is symptomatic through increasing acetylcholine levels in parts of the brain essential for memory functions.

America250 Launches New Nationwide Student Contest: ‘America’s Field Trip’

Retrieved on: 
Monday, March 4, 2024
Primary, Secondary, Education, White House, Federal Government, Public Policy, Government, Consumer, Teens, Parenting, Andrew Mellon, Federal Reserve Bank, National Park Service, BNY Mellon, Bank, National archives, Dream, Growth, First Prize, Library, Culture, Natural history, Corporate Affairs, Volunteering, Field trip, D.C, Student, Congress, Interpretation, Heart, Discovery, Inc., History, Mellon, Travel, Tourism

America’s Field Trip invites elementary, middle, and high school students to submit artwork, videos, and essays reflecting on what America means to them.

Key Points: 
  • America’s Field Trip invites elementary, middle, and high school students to submit artwork, videos, and essays reflecting on what America means to them.
  • “We’re proud to launch this nationwide effort alongside BNY Mellon, whose support will make it possible to send students on experiences of a lifetime.
  • "We are proud to partner with America250 to deepen curiosity about our nation's history," said Amy Nakamoto, General Manager of Corporate Partnerships at Discovery Education Experience.
  • "As we approach this important milestone, we are excited to work with America250 and BNY Mellon to bring this national contest to life for students nationwide."

Gilead to Present Late-Breaking Data and Real-World Evidence Highlighting Innovative Antiviral Portfolio and Research Pipeline at CROI 2024

Retrieved on: 
Monday, February 26, 2024
Biotechnology, FDA, AIDS, Health, Pharmaceutical, Research, Infectious Diseases, Hospitals, Science, COVID-19, Clinical Trials, Senior, Hepatitis B, CROI, Hook effect, RWE, STR, Viral, Virus, DTG, Mortality, Lenacapavir, Hepatitis, HBV, Vesatolimod, Fungal infection, HIV-1, MDR, PWH, HDV, Boxed warning, European, Islatravir, Tablet, European Commission, Liver disease, Omicron, Conference, Safety, ART, Civil service commission, Abstract, Bictegravir, Natural history, Virology, Emtricitabine, Gilead Sciences, MD, Food, Coinfection, Immunodeficiency, European Economic Area, Risk, Prevalence, Dexamethasone, Patient, Tuberculosis, INSIGHT, HIV, Pharmaceutical industry

Outcomes from pipeline research studies will also provide insights into investigational treatment candidates, including the novel combination regimen of lenacapavir plus bictegravir.

Key Points: 
  • Outcomes from pipeline research studies will also provide insights into investigational treatment candidates, including the novel combination regimen of lenacapavir plus bictegravir.
  • Additionally, a late-breaker oral presentation of Week 24 data from the INSIGHT trial evaluating Biktarvy in people with HIV and tuberculosis will be presented.
  • Gilead will present new data evaluating the safety and efficacy of Hepcludex® (bulevirtide) in people living with the coinfections of HIV, hepatitis B (HBV) and hepatitis delta (HDV).
  • Biktarvy combines the novel, unboosted INSTI bictegravir, with the Descovy® (emtricitabine 200 mg/tenofovir alafenamide 25 mg tablets, F/TAF) backbone.

Patient-Reported Outcomes Data from Longest Running Dermatology Study: TARGET-DERM AA Poster Presented at American Academy of Dermatology (AAD) 2024

Retrieved on: 
Friday, March 8, 2024
Alopecia areata, Anxiety, Natural history, Psoriasis, Autoimmune disease, Professor, Assistant, Hair loss, Quality of life, Atopic dermatitis, Alopecia totalis, Skin, Mental, Entrepreneurship, RWE, Hashimoto's thyroiditis, AA, AAD, Vitiligo, University, Hidradenitis suppurativa, Patient, Disease, Dermatitis, Diagnosis, Social stigma, Depression, Nursing

DURHAM, N.C., March 8, 2024 /PRNewswire/ -- New patient-reported outcomes data presented at AAD 2024 from Target RWE's TARGET-DERM AA registry confirms the high unmet need and significant disease burden in patients with moderate to severe alopecia areata (AA). TARGET-DERM is the largest and most comprehensive longitudinal RWE study observing patients with immune-mediated inflammatory skin conditions in the United States.

Key Points: 
  • Patients with AA have a high unmet medical need due to comorbid conditions and high impact on quality of life.
  • The most common comorbid conditions associated with AA include vitiligo, atopic dermatitis (eczema), Hashimoto's thyroiditis, psoriasis, and systemic lupus erythematosus.
  • TARGET-DERM AA is distinctively positioned to address real-world access to care, disease progression, diagnosis, and treatment in patients with moderate to severe AA.
  • "What's important about our data presented at AAD and TARGET-DERM AA is that we are gaining insights on the impact of the disease.

Avidity Biosciences Announces Positive AOC 1001 Long-term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 HARBOR™ Trial

Retrieved on: 
Monday, March 4, 2024
Therapy, Division, PD, PRO, Natural history, Doctor of Philosophy, Myotonic dystrophy, MD, Friends, Neurology, Hope, Neuromuscular medicine, Conference, DM1, Facial muscles, Safety, Muscular Dystrophy Association, Biomarker, RNA, Webcast, Patient, Elbow, Family, Disease, QMT, Pediatrics, AOC, Publication, MDA, Volume Eight, Episcopal conference, Partnership, Pharmaceutical industry

SAN DIEGO, March 4, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE™) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.

Key Points: 
  • These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1.
  • Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.
  • Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran.
  • ET to discuss new positive long-term del-desiran (AOC 1001) data from the MARINA-OLE™ trial in people living with DM1.

Mitsubishi Tanabe Pharma America to Showcase Presentations on ALS Research at 2024 MDA Clinical & Scientific Conference

Retrieved on: 
Friday, March 1, 2024
Real-World Evidence, Massachusetts General Hospital, Spirometry, Data mart, ALS, Doctor of Philosophy, Interim analysis, Edaravone, M.S, Hospital, Conference, Natural history, State university system, Abstract, CDM, SVC, MDA, Phase 4, Episcopal conference, MTPA, Pharmaceutical industry

JERSEY CITY, N.J., March 1, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of five abstracts in amyotrophic lateral sclerosis (ALS) at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held in Orlando, Fla., March 3-6.

Key Points: 
  • JERSEY CITY, N.J., March 1, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of five abstracts in amyotrophic lateral sclerosis (ALS) at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held in Orlando, Fla., March 3-6.
  • "These presentations reinforce our commitment to making a positive impact on the ALS community by better understanding disease markers and outcomes and providing important information on our treatment options for clinical practice."
  • Interim Analysis of the Radicava/Edaravone Findings in Biomarkers from ALS (REFINE-ALS) Study (James Berry, M.D., M.P.H.
  • Real-World Evidence on Treatment Retention, Safety, and Tolerability of Edaravone in Canadian Patients With Amyotrophic Lateral Sclerosis (Dung Pham, PhD; MTPA-CA)

Mallinckrodt Announces U.S. FDA Approval of Supplemental New Drug Application for Acthar® Gel (repository corticotropin injection) Single-Dose Pre-filled SelfJect™ Injector

Retrieved on: 
Friday, March 1, 2024
Chorioretinitis, Natural history, Caregiver, New Drug Application, Ankylosing spondylitis, Optic neuritis, Education, FDA, Food, Adnexa, Shanda, Uveitis, Eye, Adrenocorticotropic hormone, Multiple sclerosis, Condylar resorption, Safety, Rheumatoid arthritis, Patient, Sclerosis, Disease, Keratitis, MRCP, Insurance, Pharmaceutical industry

DUBLIN, March 1, 2024 /PRNewswire/ -- Mallinckrodt plc, a global specialty pharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has approved Mallinckrodt's supplemental New Drug Application (sNDA) for the Acthar® Gel (repository corticotropin injection) Single-Dose Pre-filled SelfJect™ Injector (herein referred to as "SelfJect"), a new delivery device for Acthar Gel for appropriate patients with a range of chronic and acute inflammatory and autoimmune conditions.2 SelfJect is intended to provide the appropriate subcutaneous dose of Acthar Gel, as prescribed by a healthcare professional, and is designed to help give patients control of their administration.1,3

Key Points: 
  • "We're excited to bring this innovation to U.S. patients with chronic and acute inflammatory and autoimmune conditions.
  • Mallinckrodt also offers a team of field-based experts who provide education for healthcare professionals on the reimbursement process as well as tools available for patients.
  • Acthar Gel will continue to be available as an injection with a vial and syringe.
  • Controlled clinical trials have shown Acthar to be effective in speeding the resolution of acute exacerbations of multiple sclerosis.

Benitec Biopharma Releases Second Quarter 2024 Financial Results and Provides Operational Update

Retrieved on: 
Tuesday, February 13, 2024
Form 10-Q, Natural history, Research, Oropharyngeal dysphagia, Oculopharyngeal muscular dystrophy, Data monitoring committee, Dysphagia, FIH, Safety, Patient, Protocol, Disorder, DSMB, Pharmaceutical industry

HAYWARD, Calif., Feb. 13, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced financial results for its second fiscal quarter ended December 31, 2023. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission.

Key Points: 
  • HAYWARD, Calif., Feb. 13, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announced financial results for its second fiscal quarter ended December 31, 2023.
  • Following the DSMB recommendation, a second subject has been scheduled to receive BB-301 in February 2024.
  • Total Expenses for the quarter ended December 31, 2023, were $6.9 million compared to $5.6 million for the quarter ended December 31, 2022.
  • The loss from operations for the quarter ended December 31, 2023, was $6.9 million compared to a loss of $5.6 million for the quarter ended December 31, 2022.